2 active not recruiting, 2 recruiting, 1 completed, 1 withdrawn. 6 Phase 2, 4 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Active not recruiting 2, Recruiting 2, Completed 1, Withdrawn 1 |
| Phase 1 | 4 | Active not recruiting 2, Recruiting 2 |
Follow Pompe Disease (Late-onset): new trials by email Literature Review of Pompe Disease (Late-onset) trials Research Report on Pompe Disease (Late-onset)
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) AB-1009 (GAA Gene) - Pompe Disease (Late-onset), Pompe Disease Late-Onset | Phase 1 / Phase 2 | Recruiting | AskBio Inc | 12 | 2026-04-15 | 2026-10-13 |
| Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug GC301 - Pompe Disease (Late-onset) | Phase 1 / Phase 2 | Recruiting | GeneCradle Inc | 33 | 2024-04-19 | 2025-07-03 |
| Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Clenbuterol, Placebos - Pompe Disease (Late-onset) | Phase 2 | Withdrawn | Duke University | 2023-01-01 | 2022-08-17 | |
| Gene Transfer Study in Patients With Late Onset Pompe Disease zocaglusagene nuzaparvovec - Pompe Disease (Late-onset) | Phase 1 / Phase 2 | Active not recruiting | Astellas Gene Therapies | 11 | 2020-10-27 | 2026-09-02 |
| A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) SPK-3006 - Pompe Disease, Pompe Disease (Late-onset) | Phase 1 / Phase 2 | Active not recruiting | Spark Therapeutics, Inc. | 4 | 2020-10-01 | 2024-11-27 |
| A Study of rhGAA in Patients With Late-Onset Pompe Disease Myozyme - Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II) | Phase 2 | Completed | Genzyme, a Sanofi Company | 5 | 2005-02-01 | 2014-02-06 |
Glycogen Storage Disease Type II (6) · Lysosomal Storage Diseases (2) · Glycogen Storage Disease (1) · Neuromuscular Diseases (1)
Brain Diseases (6) · Brain Diseases, Metabolic (6) · Brain Diseases, Metabolic, Inborn (6) · Carbohydrate Metabolism, Inborn Errors (6) · Central Nervous System Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Genetic Diseases, Inborn (6) · Lysosomal Storage Diseases, Nervous System (6) · Metabolic Diseases (6) · Metabolism, Inborn Errors (6) · Nervous System Diseases (6) · Nutritional and Metabolic Diseases (6) · Glycogen Storage Disease (5) · Lysosomal Storage Diseases (4)
Clenbuterol (1) · GAA protein, human (1) · alpha-Glucosidases (1)
AskBio Inc (1) · Astellas Gene Therapies (1) · Duke University (1) · GeneCradle Inc (1) · Genzyme, a Sanofi Company (1) · Spark Therapeutics, Inc. (1)
United States (4) · Netherlands (2) · United Kingdom (2) · Canada (1) · China (1) · Denmark (1) · France (1) · Germany (1) · Italy (1)
Dwight Koeberl, MD, PhD (1) · Guang Yang (1) · Tahseen Mozaffar, MD (1)
2019 (2)